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Personalised Prophylaxis in a Child with Haemophilia A and Type 1 Diabetes

Maria Sol Cruz1, Josefina Santillan1, Julieta Lesser1

  • 1Hemophilia Foundation of Salta, Salta 4400, Argentina.

Clinics and Practice
|June 2, 2021
PubMed

Insights

This case study highlights personalized treatment for a child with severe hemophilia A and type 1 diabetes. Switching to recombinant factor VIII (rFVIII) improved adherence and prevented bleeds, demonstrating effective management of coexisting conditions.

Area of Science:

  • Pediatric Hematology
  • Endocrinology
  • Case Study

Background:

  • Poor management of type 1 diabetes or hemophilia A can cause severe complications.
  • Concomitant diagnosis of severe hemophilia A and type 1 diabetes presents unique challenges in pediatric care.

Purpose of the Study:

  • To describe the management of a pediatric patient with both severe hemophilia A and type 1 diabetes.
  • To evaluate the efficacy and tolerability of switching to a recombinant factor VIII (rFVIII) product.

Main Methods:

  • A case report of an 8-year-old boy with severe hemophilia A and type 1 diabetes.
  • Transition of hemophilia A treatment from plasma-derived FVIII to simocotocog alfa (rFVIII).
  • Monitoring for bleeds, joint damage, and adverse events over seven years of prophylaxis.

Main Results:

  • Personalized treatment with rFVIII allowed a reduced dosing schedule (twice weekly).
  • The patient experienced no spontaneous bleeds and no joint damage after over seven years of prophylaxis.
  • rFVIII was well tolerated with no observed treatment-related adverse events.

Conclusions:

  • Personalized treatment strategies are crucial for pediatric patients with comorbid conditions like hemophilia A and type 1 diabetes.
  • Switching to rFVIII facilitated adherence and provided effective prophylaxis, preventing complications.
  • This case underscores the benefits of individualized care in managing complex pediatric hematological and endocrine disorders.

Keywords:
coagulation factor VIIIdiabeteshaemophilia Apersonalised prophylaxissimoctocog alfa

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